CORAL GABLES, Fla.(BUSINESS WIRE)
Catalyst Pharmaceutical Partners, Inc. announced today it has begun the
process required to establish an expanded access program (EAP) to make
available in the United States its investigational potassium channel
inhibitor, Firdapse? (amifampridine phosphate or 3,4-DAP phosphate), to
patients diagnosed with Lambert-Eaton
Myasthenic Syndrome (LEMS) through their neuromuscular disease
specialists. The product, currently in Phase 3 development, will be
provided at no cost until sometime after approval. Program enrollment
will begin in the next few months, pending FDA review and approvals.
As part of our ongoing commitment to the LEMS community, we are making
Firdapse available to those who would like access to this
investigational drug, but are unable to participate in clinical studies
or travel to sites where it is available, said Patrick J. McEnany, CEO
of Catalyst Pharmaceutical Partners, Inc.
Patients diagnosed with LEMS or physicians treating such patients can
call toll free 1-844-FIRDAPSE (1-844-347-3277) to inquire about this
In previously published clinical trials, amifampridine has been shown
to reduce neuromuscular weakness and symptoms of autonomic dysfunction
in LEMS patients while being well tolerated, said Shin J. Oh, MD,
Distinguished Professor Emeritus of Neurology, University of Alabama at
Birmingham School of Medicine. I am pleased that the company will be
able to make its drug available to patients, who need it, while
diligently pursuing FDA approval.
Expanded access is a mechanism supported by regulatory agencies for
getting investigational treatment to patients who have a life
threatening or severely debilitating disease and who cannot be
satisfactorily treated with an alternative therapy approved by the FDA.
Expanded access programs are not required by regulatory agencies. These
programs are initiated and supported by drug manufacturers in
recognition of the promise an unapproved drug may hold for patients
facing a life-threatening or severely debilitating disease.
About Lambert-Eaton Myasthenic Syndrome (LEMS)
LEMS is an autoimmune neuromuscular disease in which the release of
acetylcholine is decreased at the neuromuscular junction, resulting in
muscle weakness. Patients with LEMS typically present with fatigue,
muscle pain and stiffness that is generally more marked in the proximal
muscles, most often muscles of the legs and trunk. Other symptoms may
include reduced reflexes, drooping eyelids, facial weakness and
swallowing problems. Patients often report a dry mouth, impotence,
constipation and light-headedness on standing. On occasion, these
problems can be life threatening when the weakness involves respiratory
muscles. Approximately 50 percent of patients diagnosed with LEMS have
an underlying malignancy, most commonly small cell lung cancer. LEMS is
therefore regarded as a paraneoplastic syndrome (a condition that arises
as a result of cancer elsewhere in the body).
Firdapse, amifampridine phosphate or 3,4-diaminopyridine phosphate
(3,4-DAP), is a potassium channel inhibitor. By blocking this ion
channel, Firdapse increases the nerve repolarization time, which causes
an increase in the influx of calcium, thereby causing more acetylcholine
to be released, restoring muscle fiber contraction thus relieving muscle
weakness caused by LEMS. In addition to LEMS, other potential orphan
neuromuscular indications for Firdapse include certain types of
Myasthenia Gravis and Congenital Myasthenic Syndrome, among others.
Firdapse has been granted orphan drug and breakthrough therapy
designations by the U.S. FDA, and orphan medicinal product designation
in the European Union for the treatment of LEMS. It is approved and
commercialized in the E.U. by BioMarin Pharmaceutical. Catalyst recently
announced it has reached its patient enrollment target for its Phase 3,
double-blind, placebo-controlled, clinical trial evaluating the safety
and efficacy of Firdapse for the treatment of LEMS. Upon completion of
the Phase 3 trial, assuming it is successful, Catalyst expects to begin
submitting a rolling new drug application with the FDA in 2015.
About Catalyst's Commitment to LEMS Patients
Catalyst is committed to bringing all patients diagnosed with LEMS a
quality, safe and effective, FDA approved product, regardless of
financial circumstances. The Company is already working to establish a
patient assistance program that will be designed to help patients
without insurance, those in Medicare Part D, or Medicaid, and those
facing financial challenges. Catalyst will work hard to understand the
needs of patients and is committed to helping them afford their
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical
company focused on the development and commercialization of prescription
drugs targeting rare (orphan) neuromuscular and neurological diseases,
including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms,
and Tourette Syndrome. In 2012, Catalyst licensed Firdapse from
BioMarin, and Catalyst assumed management of the Phase 3 pivotal trial,
which was initiated by BioMarin. Firdapse is the first and only European
approved drug for symptomatic treatment in adults with LEMS.
Catalyst is also developing a potentially safer and more potent
vigabatrin analog (designated CPP-115) to treat infantile spasms, and
epilepsy, as well as other neurological conditions associated with
reduced GABAergic signaling, like post-traumatic stress disorder and
Tourette syndrome. CPP-115 has been granted U.S. orphan drug designation
for the treatment of infantile spasms by the FDA and has been granted
E.U. orphan medicinal product designation for the treatment of West
Syndrome by the European Commission. For more information, please visit www.catalystpharma.com.
This press release contains forward-looking statements.
Forward-looking statements involve known and unknown risks and
uncertainties, which may cause Catalyst's actual results in future
periods to differ materially from forecasted results. A number of
factors, including the anticipated timing of the receipt of top-line
results from the double-blind, placebo-controlled portion of the Phase 3
trial of Firdapse, whether historic metrics of patients enrolled in the
trial who complete the run-in phase of the trial and are randomized into
the double-blind, placebo-controlled portion of the trial will continue
to apply, such that at least 36 patients will be randomized into the
double-blind, placebo-controlled portion of the trial from the patients
already enrolled in the trial, whether the Phase 3 trial will be
successful, whether the receipt of breakthrough therapy designation for
Firdapse will expedite the development and review of Firdapse by the FDA
or the likelihood that the product will be found to be safe and
effective, whether an NDA for Firdapse will ever be accepted for filing
by the FDA, the timing of any such NDA filing or acceptance, whether
Catalyst will be the first company to receive an approval for 3,4-DAP,
giving it 7-year marketing exclusivity for its product, whether any of
Catalyst's product candidates will ever be approved for
commercialization or successfully commercialized, and those other
factors described in Catalyst's Annual Report on Form 10-K for the
fiscal year 2013 and its other filings with the U.S. Securities and
Exchange Commission (SEC), could adversely affect Catalyst. Copies of
Catalyst's filings with the SEC are available from the SEC, may be found
on Catalyst's website or may be obtained upon request from Catalyst.
Catalyst does not undertake any obligation to update the information
contained herein, which speaks only as of this date.
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LaVoie Health Science
Connolly or , 617-374-8800
Catalyst Pharmaceutical Partners, Inc.
Chief Executive Officer
Source: Catalyst Pharmaceutical Partners, Inc.